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Clinical Study on the Safety and Efficacy of PID23 Injection in Patients With Relapsed/Refractory Acute Leukemia (R/R AL)

2026년 7월 9일 업데이트: Zhujiang Hospital

A Single-center, Single-arm, Open-label Clinical Study on the Safety and Efficacy of PID23 Injection in Treating Patients With Relapsed/Refractory Acute Leukemia

Relapsed or refractory acute leukemia (R/R AL) is a life-threatening blood cancer with poor outcomes and limited treatment options. PID23 Injection is an innovative in vivo CAR-T therapy that delivers a viral vector encoding three targets (CD19, BCMA, and CD70) directly into patients, enabling their own T cells to generate functional CAR-T cells against leukemia cells. This single-center, single-arm, open-label, dose-escalation study (3 dose levels: 0.8×10⁹, 2×10⁹, and 4×10⁹ TU) plans to enroll 3-18 patients with R/R AL aged 3-75 years, ECOG 0-2, and positive for at least one target. The primary objective is to evaluate safety, tolerability, and determine the recommended dose. Secondary objectives include preliminary efficacy (remission, survival), pharmacokinetics (CAR-T expansion), pharmacodynamics (cytokine changes), and exploratory viral clearance. After a single intravenous infusion, patients are hospitalized for ≥3 weeks, followed by monthly visits for 3 months, then every 3 months for up to 2 years. Enrollment is from May 2026 to May 2027, with follow-up through May 2029. The study is conducted at Zhujiang Hospital of Southern Medical University (PI: Prof. Li Yuhua).

연구 개요

연구 유형

중재적

등록 (추정된)

18

단계

  • 초기 1단계

연락처 및 위치

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연구 연락처

연구 장소

    • Guangdong
      • Guangzhou, Guangdong, 중국, 510282

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

  • 어린이
  • 성인
  • 고령자

건강한 자원 봉사자를 받아들입니다

아니

설명

Inclusion Criteria:

  1. Patient or legal guardian voluntarily signs the informed consent form (ICF), demonstrating understanding of the study purpose and procedures, and willingness to participate.
  2. Age 3 to 75 years, inclusive, male or female.
  3. Diagnosis of relapsed or refractory acute leukemia per guideline criteria:

Relapsed: reappearance of leukemic cells in peripheral blood or bone marrow blasts ≥5% after achieving complete remission (CR); Refractory: failure to achieve CR after 2 courses of standard induction chemotherapy; relapse within 12 months after consolidation/intensification therapy; relapse after 12 months with no response to conventional chemotherapy; 2 or more relapses; extramedullary leukemia relapse or persistence; relapse after allogeneic hematopoietic stem cell transplantation.

4.Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2. 5.Life expectancy ≥12 weeks. 6.Bone marrow morphology showing ≥5% primitive/immature lymphocytes (blasts). 7.Tumor cells positive for CD19, BCMA, or CD70 expression by flow cytometry. 8.Adequate major organ function, defined as:

  1. Cardiac: left ventricular ejection fraction (LVEF) ≥40% by echocardiogram;
  2. Renal: serum creatinine ≤2.0× upper limit of normal (ULN), or creatinine clearance ≥50 mL/min (Cockcroft-Gault formula);
  3. Hepatic: ALT and AST ≤3.0×ULN (≤5.0×ULN if with liver involvement); total bilirubin ≤2.0×ULN (≤3.0×ULN for Gilbert's syndrome);
  4. Pulmonary: oxygen saturation ≥92% on room air;
  5. Hematologic: absolute neutrophil count (ANC) ≥1.0×10⁹/L, platelets ≥50×10⁹/L, hemoglobin ≥80 g/L (with bone marrow involvement, ANC ≥0.5×10⁹/L, platelets ≥20×10⁹/L permitted) - assessments allowed after transfusion or hematopoietic growth factor support.

9. For women of childbearing potential, negative serum pregnancy test; all participants agree to use reliable (non-rhythm) contraceptive methods from ICF signing through 1 year post-PID23 infusion.

Exclusion Criteria:

  1. Prior treatment with CAR-T or other genetically modified cell therapies, unless the investigator determines that safety risks have been adequately excluded.
  2. Received the following anti-tumor therapies prior to PID23 infusion: Chemotherapy or molecular targeted therapy within 14 days or 5 half-lives (whichever is longer) (excluding conditioning chemotherapy and intrathecal chemotherapy; intrathecal therapy must be stopped ≥1 week prior to PID23 infusion); Radiotherapy to non-hematopoietic sites within 7 days; Radiotherapy to hematopoietic sites within 14 days.
  3. Any of the following cardiac conditions:

(1) New York Heart Association (NYHA) Class III or IV congestive heart failure; (2) Myocardial infarction or coronary artery bypass grafting (CABG) within 6 months prior to enrollment; (3) Clinically significant ventricular arrhythmia, or unexplained syncope (excluding vasovagal or dehydration-related); (3) History of severe non-ischemic cardiomyopathy. 4.Active or uncontrolled infection requiring systemic therapy within 1 week prior to screening.

5.Grade 2-4 acute graft-versus-host disease (GVHD) or moderate-to-severe chronic GVHD within 4 weeks prior to screening.

6.Cerebrovascular accident or seizure within 6 months prior to screening. 7.Deep vein or arterial thrombosis event within 6 months prior to screening. 8.Active malignancy other than acute leukemia (excluding: inactive disease with treatment completed >2 years; adequately treated cervical carcinoma in situ, basal/squamous cell skin carcinoma, localized prostate cancer post-curative surgery, ductal carcinoma in situ post-curative surgery).

9.Received (attenuated) live vaccine within 4 weeks prior to screening. 10.Any other condition that, in the investigator's judgment, makes the patient unsuitable for participation in this study.

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 해당 없음
  • 중재 모델: 단일 그룹 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: PID23 Injection (Dose Escalation)
Participants receive a single intravenous infusion of PID23 Injection at one of three dose levels (0.8×10⁹, 2×10⁹, or 4×10⁹ TU) according to a rapid titration + standard 3+3 dose-escalation design. The starting dose is 0.8×10⁹ TU, with escalation to higher dose levels guided by safety and pharmacokinetic assessments. All subjects are hospitalized for observation for at least 3 weeks post-infusion and followed for up to 2 years.
PID23 is an in vivo CAR-T product, a lentiviral vector encoding CD19, BCMA, and CD70 chimeric antigen receptors, administered as a single intravenous infusion

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Incidence of Dose-Limiting Toxicities (DLTs)
기간: Up to 21 days post-PID23 infusion
Number of participants experiencing DLTs during the DLT observation period. DLT is defined as any treatment-emergent adverse event meeting protocol-specified severity criteria assessed per CTCAE v6.0 and ASTCT criteria for CRS/ICANS.
Up to 21 days post-PID23 infusion
Incidence and Severity of Treatment-Emergent Adverse Events
기간: Up to 2 years post-PID23 infusion
Number and percentage of participants experiencing adverse events (AEs), graded per CTCAE v6.0. Includes all AEs, serious AEs (SAEs), and AEs of special interest (CRS, ICANS, HLH/MAS). Causality assessment performed by the investigator.
Up to 2 years post-PID23 infusion

2차 결과 측정

결과 측정
측정값 설명
기간
Objective Response Rate at 3 Months
기간: At 3 months post-infusion
Proportion of participants achieving complete remission (CR) or CR with incomplete blood count recovery (CRi) at 3 months post-infusion. CR defined as bone marrow blasts <5%, no peripheral blasts, ANC ≥1.0×10⁹/L, platelets ≥100×10⁹/L, no extramedullary disease. CRi defined as meeting all CR criteria except platelet <100×10⁹/L and/or ANC <1.0×10⁹/L.
At 3 months post-infusion
Duration of Remission (DOR)
기간: Up to 2 years post-infusion
Time from first documented CR or CRi to first documented disease progression (PD) or death from any cause, whichever occurs first. Participants who do not achieve CR/CRi are not applicable for this analysis. Assessed per protocol until 2 years post-infusion.
Up to 2 years post-infusion
Progression-Free Survival (PFS)
기간: Up to 2 years post-infusion
Time from PID23 infusion to first documented disease progression (PD) or death from any cause, whichever occurs first. Participants alive without progression are censored at the date of last disease assessment. Assessed per protocol through 2 years post-infusion or until event.
Up to 2 years post-infusion
Overall Survival (OS)
기간: Up to 2 years post-infusion
Time from PID23 infusion to death from any cause. Participants alive at the end of follow-up are censored at the date of last known survival status. Assessed through 2 years post-infusion or until death, whichever occurs first.
Up to 2 years post-infusion
Change from Baseline in Bone Marrow Blast Percentage
기간: Baseline through 2 years post-infusion
Change from baseline in percentage of bone marrow blasts assessed by bone marrow morphology. Measured at screening and at protocol-specified follow-up time points (M1, M2, M3, and every 3 months thereafter). Assessed per protocol schedule.
Baseline through 2 years post-infusion
Maximum Concentration (Cmax) of CAR-T Cells in Peripheral Blood
기간: Day 0 through 2 years post-infusion
Maximum concentration (Cmax) of CAR-T cells in peripheral blood, measured as CAR DNA copy number by quantitative PCR (copies/μg DNA) and/or percentage of CAR+ cells by flow cytometry. Blood samples collected at protocol-specified time points: 0h, 30min, 2h, 6h, 12h, D2, D4, D7, D10, D14, D21, M1, M2, M3, and long-term follow-up. Samples analyzed by the technology partner.
Day 0 through 2 years post-infusion
Time to Maximum Concentration (Tmax) of CAR-T Cells
기간: Day 0 through 2 years post-infusion
Time to reach maximum concentration (Tmax) of CAR-T cells in peripheral blood, measured as CAR DNA copy number by quantitative PCR. Blood samples collected at protocol-specified time points from immediately post-infusion through long-term follow-up. Tmax determined from the concentration-time profile.
Day 0 through 2 years post-infusion
Area Under the Curve (AUC0-28d) of CAR-T Cells
기간: Day 0 through Day 28
Area under the concentration-time curve from Day 0 to Day 28 (AUC0-28d) of CAR-T cells in peripheral blood, measured as CAR DNA copy number by quantitative PCR. Calculated using the linear trapezoidal rule. Blood samples collected at protocol-specified time points through Day 28.
Day 0 through Day 28
Change from Baseline in Serum Cytokine Levels
기간: Baseline through Month 1
Change from baseline in serum cytokine concentrations, including but not limited to IL-6, IL-1β, IL-2/IL-2R, IL-8, IL-10, TNF-α, IFN-γ, CRP, and ferritin. Blood samples collected at protocol-specified time points: screening, D0 (pre-infusion), D2, D4, D7, D10, D14, and M1.
Baseline through Month 1

기타 결과 측정

결과 측정
측정값 설명
기간
Viral Clearance from Body Fluids
기간: Day 0 through Month 1 or until two consecutive negatives, whichever came first
Detection of viral vector sequences in body fluid samples including blood, saliva, urine, and stool. Viral clearance is defined as two consecutive negative results. Samples collected at protocol-specified time points: 0h, 30min, 2h, 6h, 12h, D2, D4, D7, D10, D14, D21, and M1. Samples may be stored at -80°C and shipped in batches to the technology partner for analysis. Viral clearance assessed until two consecutive negative results are obtained.
Day 0 through Month 1 or until two consecutive negatives, whichever came first

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (추정된)

2026년 8월 1일

기본 완료 (추정된)

2029년 5월 30일

연구 완료 (추정된)

2030년 3월 31일

연구 등록 날짜

최초 제출

2026년 7월 6일

QC 기준을 충족하는 최초 제출

2026년 7월 9일

처음 게시됨 (실제)

2026년 7월 10일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 7월 10일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 7월 9일

마지막으로 확인됨

2026년 7월 1일

추가 정보

이 연구와 관련된 용어

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

아니요

IPD 계획 설명

IPD will not be shared due to: (1) ongoing study with long-term follow-up through 2 years post-infusion; (2) small sample size (3-18) with high participant re-identification risk; (3) sensitive genetic information (CAR-T viral vector integration data) contained in the IPD; and (4) lack of infrastructure and funding for IPD sharing at this single-center investigator-initiated study. Comprehensive study findings will be published in peer-reviewed journals. Requests for access to raw IPD for legitimate research purposes may be directed to the corresponding author via email.

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

아니

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

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